Clinical studies fall into two main categories – observational and interventional. An observational study is non-experimental. Researchers observe the effects of a risk factor, such as an inherited gene, or air quality, or a treatment that has already been applied. Researchers have no control over the variables. Such a study can show a correlation between the variable and the observed outcome but cannot prove that one caused the other. In an interventional study, such as a clinical trial, researchers introduce an intervention, such as a drug treatment, exercise, or meditation and then observe the results. A rigorous form of an interventional trial divides people into two or more groups, one receiving the intervention and, for example, the other receiving a placebo or standard treatment. Both types of study are valuable, and sometimes the findings from an observational study can suggest a hypothesis and lead investigators to design a clinical trial.
The Parkinson’s Foundation’s PDGENEration: Mapping the Future of Parkinson’s Disease is a flagship initiative providing free genetic testing and genetic counseling to look at specific Parkinson’s-related genes and connect their presence with disease course, medication usage, and other outcomes. Besides potentially helping people with Parkinson’s manage their disease better, the results may empower clinical trial design, such as seeing how a medication may affect the disease depending on what type of gene is present. Dr. Tanya Simuni, Chief of Movement Disorders at the Northwestern University Feinberg School of Medicine in Chicago and Dr. Michael Schwarzschild, director of the Molecular Neurobiology Laboratory at Massachusetts General Hospital in Boston, discuss some aspects of clinical studies in PD and why it is important for people with PD to participate in them. Both institutions are Parkinson’s Foundation Centers of Excellence.
- About PD GENEration
- Fact Sheet: PDGENEration
- Episode 73: Genetics and PD – What do we know so far? (podcast)
- Episode 67: PDGENE – Genetic Counseling (podcast)
- Moving Day: A Walk for Parkinson’s Virtual Experience
About This Episode
Released: April 21, 2020
Michael Schwarzschild, MD, PhD
Dr. Schwarzschild is a neurologist at Massachusetts General Hospital and Julieanne Dorn Professor of Neurology at Harvard Medical School. With an undergraduate foundation in biochemistry he pursued a doctoral thesis on the neurochemistry of tyrosine hydroxylase, the enzyme controlling dopamine biosynthesis. After neurology residency and Parkinson's disease (PD) fellowship training at MGH he developed a translational research program focusing on the role of purines — adenosine, caffeine and urate — among environmental and genetic influences in animal models and clinical studies of PD. He directs the Molecular Neurobiology Lab at the MassGeneral Institute for Neurodegenerative Disease where his interdisciplinary research program spans the neurobiology, epidemiology and clinical science of Parkinson’s. His group is currently focused on the development of clinical trials to prevent PD, building off recent advances in identifying prodromally and genetically at-risk populations. In 2018 he was elected to a 2nd six-year term leading the Parkinson Study Group (PSG), a consortium of North American clinical trial sites and investigators dedicated to finding improved treatments for people with Parkinson’s. At MGH he cares for patients with PD and their families in a weekly movement disorders clinic.
Tanya Simuni, MD
Dr. Simuni graduated from Leningrad Medical School and completed an internship in medicine in Leningrad, Russia. She subsequently completed an internship in internal medicine at Albert Einstein Medical Center and a neurology residency and a clinical neurophysiology fellowship at Temple University in Philadelphia, Pennsylvania. She then pursued a movement disorders fellowship at the University of Pennsylvania, where she served on the clinical faculty of the Department of Neurology for three years and held the position of Medical Co-Director of the Parkinson’s disease and Movement Disorders surgical program.
Dr. Simuni joined the faculty of the Northwestern University in 2000 to build and lead a multidisciplinary movement disorders center that is recognized by the Parkinson’s Foundation and Wilson’s Foundation and serves as a training model in the region. She is the lead investigator of a number of clinical trials on experimental pharmacology, non-motor manifestations, and pharmacological management of PD. She is currently the Investigator of more than a dozen clinical trials. She serves on a number of Steering Committees for the PD national clinical trials, several committees of the Parkinson Study Group and the Parkinson’s Foundation. She is the Site PI and serve on the Steering Committee for the largest PD biomarker initiative funded by the MJFF (PPMI study). Dr. Simuni is the site PI for the Network for Excellence in Neuroscience Clinical Trials (NEXT) Northwestern Clinical Site (U10). She has more than one hundred publications in peer-reviewed scientific journals and she has lectured nationally and internationally on movement disorders.
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